MICA: Childhood Arthritis Response to Treatment consortium - partnership to define stratified medicine tools for childhood inflammatory arthritis

MICA：儿童关节炎治疗联盟 - 合作定义儿童炎症性关节炎的分层医学工具

• 批准号: MR/M004600/1
• 负责人: Lucy Wedderburn
• 金额: $ 50.99万
• 依托单位: University College London
• 依托单位国家: 英国
• 项目类别: Research Grant
• 财政年份: 2014
• 资助国家: 英国
• 起止时间: 2014 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=MR%2FM004600%2F1
• 关键词: MICA; Childhood; Arthritis; Response; Treatment

Arthritis (swelling, pain and inflammation of the joints) affects 1 in 1000 children and young people. Arthritis of children and young people is called juvenile idiopathic arthritis (JIA). If JIA is not fully treated it causes severe pain, joint restriction swelling and stiffness, fatigue, poor growth, thinning of bones, time off school, reduced social and sporting activities and lost family life opportunities. JIA can lead to major disability that lasts well into adult life. There are many modern, powerful medicines that can be used to treat JIA better than before. However we do not yet have reliable and child-friendly tests that can predict which drug will work best for each child. Each of these drugs may also have serious side effects. At the moment, children and young people (and their families) have to wait after starting a new drug to see if it works, and whether any important side effects happen. If it does not work, or side effects crop up, only then are they allowed to try another. This "lets try it and see" approach takes time and exposes children to drugs which might not work well, yet often have important short and long term side effects. Therefore there is an urgent need to develop ways that can help to predict how an individual child will respond to each drug, and so allow children with JIA to be treated with the most appropriate medicine. This approach is now known as 'precision' or 'stratified' medicine. Early control of the arthritis allows children to return to their education, as well as full sporting and family life activities, whilst continued control of arthritis prevents disability and avoids lost opportunities in the long-term.At the moment, four major UK studies are collecting valuable data about response to treatment from children and young people with JIA across the country. Between them, these studies have a lot of data, both clinical as well as laboratory data, that can address this need. In this partnership we propose to bring these studies together to form a unique Consortium, called the Childhood Arthritis Response to Treatment Consortium (CHART). CHART will work with all the main national and international stakeholders including clinical and research collaborators, as well as families and patients who lives are affected by JIA. The overall aim of CHART is to directly improve clinical care for children with arthritis. The key aspects of the work planned in the CHART Consortium are to : 1. Find out exactly what data and samples are already available in the four major UK studies of JIA 2. Design and agree a way to share all of the data, and bring the data together 3. Agree to collect data and samples for understanding response to treatment in the same way in the future, in order to speed up progress for all children with JIA4. Give many more children and families the opportunity to contribute to these studies by including new centres and collaboratorsThe CHART Consortium, if established, will be one of the largest of its kind in the world. The CHART researchers will have the common goal of making treatment of JIA more individualised and precise, to help every child with arthritis return to a full normal life as quickly as possible.

关节炎（关节肿胀，疼痛和炎症）影响1/1000的儿童和青少年。儿童和青少年的关节炎称为幼年特发性关节炎（JIA）。如果JIA没有得到充分的治疗，它会导致严重的疼痛，关节限制肿胀和僵硬，疲劳，生长不良，骨骼变薄，辍学，减少社会和体育活动，失去家庭生活的机会。JIA可能导致严重残疾，并持续到成年。有许多现代，强大的药物，可以用来治疗贾比以前更好。然而，我们还没有可靠和儿童友好的测试，可以预测哪种药物对每个孩子最有效。这些药物中的每一种都可能有严重的副作用。目前，儿童和年轻人（以及他们的家人）在开始使用新药后必须等待，看看它是否有效，以及是否会发生任何重要的副作用。如果它不起作用，或者出现副作用，只有这样他们才被允许尝试另一种。这种“让我们试试看”的方法需要时间，并使儿童接触可能不起作用的药物，但往往有重要的短期和长期副作用。因此，迫切需要开发有助于预测单个儿童对每种药物的反应的方法，从而使JIA儿童得到最合适的药物治疗。这种方法现在被称为“精确”或“分层”医学。关节炎的早期控制可以让孩子们回到他们的教育，以及充分的体育和家庭生活活动，而关节炎的持续控制可以防止残疾，并避免失去长期的机会。目前，四个主要的英国研究正在收集有价值的数据，从全国各地的儿童和青少年与JIA的治疗反应。这些研究有大量的数据，包括临床和实验室数据，可以满足这一需求。在这种伙伴关系中，我们建议将这些研究结合在一起，形成一个独特的联盟，称为儿童关节炎治疗反应联盟（CHART）。CHART将与所有主要的国家和国际利益相关者合作，包括临床和研究合作者，以及受JIA影响的家庭和患者。CHART的总体目标是直接改善关节炎儿童的临床护理。CHART联盟计划的工作的主要方面是：1。找出确切的数据和样本已经在四个主要的英国研究JIA 2。设计并商定一种共享所有数据的方法，并将数据汇集在一起3。同意收集数据和样本，以便在未来以同样的方式了解对治疗的反应，以加快所有JIA儿童的进展4。通过包括新的中心和合作者，让更多的儿童和家庭有机会为这些研究做出贡献。CHART研究人员的共同目标是使JIA的治疗更加个性化和精确，以帮助每一个关节炎儿童尽快恢复正常生活。

项目成果

Development and implementation of 'A guide to PPIE-Early Integration into Research Proposals' in a multi-disciplinary consortium.

在多学科联盟中制定和实施“PPIE 早期融入研究计划指南”。

• DOI: 10.1093/rheumatology/kead482
• 发表时间: 2023
• 期刊: Rheumatology (Oxford, England)
• 作者: Beesley R

P168 An enriched population of tissue-resident CD8 memory T cells in young people with juvenile idiopathic arthritis recapitulate findings from mouse models of inflammatory arthritis flares

P168 患有幼年特发性关节炎的年轻人体内丰富的组织驻留 CD8 记忆 T 细胞群概括了炎症性关节炎发作小鼠模型的发现

• DOI: 10.1093/rheumatology/keac133.167
• 发表时间: 2022
• 期刊: Rheumatology
• 影响因子: 5.5
• 作者: Bolton C

CAPTURE JIA Patient Status Dataset: a consensus-derived core dataset to improve clinical care for children and young people with JIA

CAPTURE JIA 患者状况数据集：基于共识的核心数据集，用于改善 JIA 儿童和青少年的临床护理

• 发表时间: 2015
• 作者: Armitt, G

Genetic feature engineering enables characterisation of shared risk factors in immune-mediated diseases.

• DOI: 10.1186/s13073-020-00797-4
• 发表时间: 2020-11-25
• 期刊: Genome medicine
• 影响因子: 12.3
• 作者: Burren OS;Reales G;Wong L;Bowes J;Lee JC;Barton A;Lyons PA;Smith KGC;Thomson W;Kirk PDW;Wallace C
• 通讯作者: Wallace C

THU0531 The Initial Treatment of Systemic Juvenile Idiopathic Arthritis: An International Collaboration Among 7 Registries:

THU0531 系统性幼年特发性关节炎的初步治疗：7 个注册机构之间的国际合作：

• DOI: 10.1136/annrheumdis-2015-eular.1613
• 发表时间: 2015
• 期刊: Annals of the Rheumatic Diseases
• 影响因子: 27.4
• 作者: Beukelman T